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Patient-reported outcomes in clinical studies of patients with hypertrophic cardiomyopathy
Nosheen Reza1, Sharlene M Day1, Anjali Tiku Owens1
1Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
Insights
Few hypertrophic cardiomyopathy clinical studies report patient-reported outcomes (PROs). More research is needed on PROs to assess new treatments for this heart condition.
Area of Science:
- Cardiology
- Clinical Research Methodology
- Patient-Reported Outcomes
Background:
- Hypertrophic cardiomyopathy (HCM) therapies aim to improve symptoms, function, and quality of life.
- Patient-reported outcomes (PROs) are crucial for assessing treatment effectiveness in HCM.
- The current landscape of PROs in HCM clinical research remains underexplored.
Purpose of the Study:
- To characterize the use of patient-reported outcomes (PROs) in registered clinical studies for hypertrophic cardiomyopathy (HCM).
- To identify trends and characteristics of HCM clinical studies that incorporate PROs.
Main Methods:
- A systematic search of ClinicalTrials.gov was conducted for studies involving HCM participants.
- PROs were identified as outcome measures using specific search terms.
- Study characteristics were compared between studies that reported PROs and those that did not.
Main Results:
- Out of 181 registered HCM studies (1987-2022), only 35 (19%) included PROs.
- Studies reporting PROs were more likely to be interventional and randomized.
- PRO reporting has increased significantly since 2007, with the Kansas City Cardiomyopathy Questionnaire being frequently used.
Conclusions:
- A small proportion of registered HCM clinical studies report PROs.
- There is a pressing need for validated, HCM-specific PRO tools to evaluate novel therapies.
- Advancing HCM treatments necessitates robust assessment of patient-centered endpoints.
Objectives:
This study aimed to characterize patient-reported outcomes (PROs) in registered clinical studies of participants with hypertrophic cardiomyopathy (HCM).
Background:
Therapy for HCM is primarily targeted toward alleviation of symptoms and improvement in function and quality of life. Yet, the contemporary landscape of PROs in HCM clinical research has not been investigated.
Methods:
ClinicalTrials.gov was queried to identify clinical studies of HCM that reported PROs as outcome measures. All studies of HCM as the disease condition were included, and PROs were identified using specific search terms in the Outcome Measures field. Study characteristics were collected and compared between those that did versus did not report PROs.
Results:
From November 1987 to February 2022, 181 studies including participants with HCM were registered on ClinicalTrials.gov. Of these, 35 (19%) included PROs as outcome measures. Studies reporting PROs were more likely to be designated as interventional (85.7% vs. 46.6%; p < 0.001) and to involve randomization (65.7% vs. 24.7%; p = 0.003) compared with those that did not report PROs. Prior to 2007, no clinical studies that reported PROs were registered in ClinicalTrials.gov; however, PRO reporting has increased over the last 15 years. Of the 66 PRO tools or domains included as outcome measures, the Kansas City Cardiomyopathy Questionnaire was the most often used.
Conclusions:
Only approximately one in five registered clinical studies of participants with HCM report PROs. As medical, percutaneous, and surgical therapies for HCM continue to advance, HCM-specific PRO tools that assess the impacts of these new treatments on meaningful patient-related endpoints are urgently needed.
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